The Rolodex - Part I

A few weeks ago I was invited to a meeting of patient/caregiver/advocates held by the Clinical Open Innovation Team at Eli Lilly and Company. Their goal was to get direct patient perspective on how clinical trials can be more patient-centric. I hope the day was as productive for them as it was energizing for me.

There were some nationally prominent e-patients there. It was easy for me to feel a little lacking and very star-struck. There was also a large contingent of Lilly staff members included and they stayed with us the whole day. I realize it’s not an easy thing to block a whole day to focus on a single topic in corporate America. It happened.

As we talked and listened, I mentioned that I have a pretty big Rolodex of ALS patients and caregivers. The day before the Lilly meeting as I tried to prepare some thoughts, I found myself wanting to reach for the Rolodex to get their opinions that I value so. Each time I wanted to pick up the phone or send an email, I got that awful feeling that you get when you realize that a trusted advisor or friend is no longer with us. ALS is a disease that provides a big Rolodex and lots of those terrible losses.

Regina Holliday was one of the participants at the meeting. I had known of her Walking Gallery project http://reginaholliday.blogspot.com/ and thought it was a wonderful idea. When I saw some of the Walking Gallery jackets in person at the Lilly meeting, I thought it was more than wonderful. It was "wow." It has an impact.

Here are more links to give you some insights into Regina and her work --
http://en.wikipedia.org/wiki/Regina_Holliday
https://www.youtube.com/watch?v=-kMy1OvDJcw

After the meeting Regina offered to paint my Rolodex. Her offer still makes me grin from ear to ear. Now it’s time to gather some information for her about a few of the ALS rockstars in my Rolodex whose wisdom I miss so.


Mom
She is truly #1 in my Rolodex and in my life. Mom was a good mother and a wonderful homemaker and a wise human being. I have a feeling that instead of a portrait that she would prefer that I include a picture of how she raised ALS awareness a few years ago on Pennsylvania Avenue in Washington – as part of the stunning Piece by Piece display. She so wanted to get the word out about ALS – a disease that outrageously is without a treatment. She left us with some timeless quotes for the fight against ALS, including "If you can’t cure me, then at least learn from me." Amen. We lost Mom to ALS in 1997.

Persevering (the online name for Rob Tison)

 

Persevering was a young father and a really smart statistician. He was smart about a lot of things. His abilities in statistics showed us that patients can sometimes find the weaknesses in peer reviewed articles and in clinical trial design. Rob was a relentless recruiter and advocate for clinical trials, and he managed to start conversations among scientists and physicians and patients and advocates who usually don’t talk to each other directly. Rob was a game-changer for the fight against ALS. We lost Persevering to ALS in 2012.

　
Happy Physicist (the online name for Ben Harris)

HP as I called him was another really smart scientist and young father who was slapped with an ALS diagnosis. He worked up until the end as a physicist on proton accelerators used in cancer therapies. He once told me that he designed equipment that took people within inches of their lives so that their cancers might be treated. He didn’t see why someone with ALS didn’t have the same right. His mantra was, "If it is done in secret, then it is done in vain." He wanted people with ALS who were trying things (and they all do) to be sure to document their data so that others might learn and the science might move forward. One morning I opened up my Wall Street Journal there was HP on the front page, written up as a citizen scientist trying to find something to treat ALS and to push the clinical trial process aggressively. He was smarter at science than most citizens and most scientists. We lost Ben to ALS a few months ago.

And in one of life’s coincidences (or perhaps a bit of kismet?), Rob, Ben, and I all had dogs named Otis.

bbzinger (the online name for Barbara Brenner)

I first e-met bbzinger during a tweet-chat led by USAToday. She was the leader of Breast Cancer Action and that was the topic of the chat. She also had recently been diagnosed with ALS. I learned so much from her about the differences between advocacy and activism and about the ways organizations need to be challenged. She was an ALS clinical trial volunteer and kept her commitment to that trial even though it didn’t seem to have fair or transparent rules for her situation. She was a very smart attorney who was driven to make a difference with the FDA and organizations and the way diseases are fought. Her blog at barbarabrenner.net also taught us much about dying. We lost bbzinger to ALS earlier this year.

Pdwyer (the online name for Pat Dwyer)

Pat Dwyer was simply a great guy. Perhaps that’s not really so simple. Pat was a husband, father, and had a commercial fishing business in Seattle. He and his wife searched and found a clinical trial for a Diaphragm Pacer with the help of Dr. Google. They became a wonderful resource for people online seeking advice on the DPS and ALS in general. Their attitude was simply that ALS picked the wrong family when it picked on theirs (yes, another one of those "simple" things that’s not so simple in real life). His wife and young-adult children and relatives and in-laws and friends carry on the fight, having lost Pat to ALS in June. At his funeral the church was literally jammed with all kinds of different people with one thing in common – they loved a great guy named Pat.


The Rolodex - Part II to follow tomorrow, New Year's Day.

Good Financial Advisors Love Clients Who Ask Questions

And good financial advisors give good accounting about what went well and what didn't go so well in the past year.

You expect a few duds in a diverse portfolio. You expect to know about them and to learn from them.

You expect complete numbers that tell you what the returns on your investments have actually been.  You have a right to know how much money went into and is now in each of your portfolio folders.

If you knew someone naive with a financial advisor who didn't like questions or who didn't give a good, detailed accounting, would you not advise him or her to run away?   Something is terribly wrong. 

We trust our ALS organizations that fund research to be very much like good financial advisors. They help us place our investments in the most promising science. We know that some things might work and many won't work, and we expect to know about them and to learn from them.

Instead of good, businesslike accounting of projects, investments, and outcomes, we get cherry-picked  public statements on "promising" results that don't give us a clear picture of the portfolio. We see selective cumulative summaries instead of specifics.  Instead of answers to our questions, we often get evasive responses, or worse yet, no responses.

Surely they would be smarter advisors and we would all be better (and bigger) investors if we had businesslike accounting of their (ergo our) research investments in the last year. Surely it would advance the science and the funding available from investors.

We Got Plenty Of Nothing

When will there be a candid, honest discussion of the CDC's ALS Registry?  Was there one at the annual meeting in July?  Is anyone looking seriously at the scope, deliverables, and effectiveness of the very expensive project?  Is there a valid reason for the cloak of secrecy?  Really?

Following is the item in a recent advocacy "listening" survey (click here) (thanks to the chapter that shared) related to the ALS Registry:

6. National ALS Registry Issue- (may include- National Office to continue support Chapter outreach and enrollment activities by providing materials, equipment, education and training; Continued funding for the National ALS Registry; Expand the number of surveys in the registry. Encourage research use of the registry.)

   

 1st /2nd/3rd/Not a top priority this year

A single tick (or not) doesn't address the considerable concerns that many of us have --

• What are the deliverables that ALSA and now MDA deliver to earn the millions that they have been granted in contracts? 
• What are the performance standards for those deliverables and have they been met? 
• Is anybody bothered by the lack of transparency over these contracts that advocates stumbled upon quite by accident? 
• What is the uptake on self-enrollment? 
• Who is questioning the scope-creep of this project? 
• Are the risk-factor surveys eliciting sufficient response to be statistically meaningful? 
• Who attended the annual meeting in July? What was discussed? Was anything challenged?
• How do we proceed and best deploy resources to make this project effective?

So far we have plenty of nothing for tens of millions of dollars.  No answers.  No meaningful status information.  No sign of responsible project management.  No transparency from organizations that were given substantial, non-competitive contracts.

Nothing is not plenty for us.

Patients, Caregivers, and Families Have Questions. You Have Experts.

My, how they have questions.  My, how you have experts.

A reported 950 people participated in the big ALS MND symposium in Milan last week.  A vast majority of them were experts in some area of ALS research or care.  Almost of them were there as part of their jobs.

The "Ask the Experts" session that was held on Wednesday was similar to that held in past years.  We're not sure who was in the room.  Three experts talked and answered a few questions.  It was interesting, but it was hardly an inviting, inclusive way to get a lot of patient and caregiver questions to the experts.

How about crowdsourcing some questions and answers next year?  Six weeks before the symposium, reach far and wide to invite PALS, CALS, and families to ask their questions.  Encourage organizations to let their PALS, CALS, and families know about an amazing opportunity.  Gather their questions.  Some will be easy, some will be difficult, some may be uncomfortable, but all will be valuable.

Next invite every one of your expert presenters to have a crack at answering the questions in writing before the symposium begins.  Multiple answers to a question would be grand.  If experts differ on answers, that would actually an interesting dimension that has been been missing from the fight against ALS.  Let's swarm on those questions, experts.  If some experts don't want to answer questions, that would be a bit revealing, too, wouldn't it?

Finally, at the big conference, a panel of experts could discuss the themes and the responses.  And please retain the written content of the questions and responses in a public place.  That will help all of the families who feel like they are reinventing wheels every day in their fights against ALS.

This format would permit every question to be asked and answered.  It would draw in more PALS and CALS and experts into a direct conversation that they are not having today.

It would be a small step toward patient-centricity at an important symposium that seems to be tone-deaf to the concept today.  It might also enlighten some of the so-called experts that the real experts in ALS are those who are living the nightmare every day.

That's My Horse!

There is a really troubling phenomenon in reports that we get from scientific meetings and publications these days.

If the presentation or report is positive, we get "we funded that research" messages from some ALS MND organizations.

In real life, we don't know if it was a $2 bet or a $2 million bet that it  placed.  A bunch of $2 bets can turn out to provide some pretty impressive advertising.

And we don't know if a not-for-profit shines kinder lighting on projects that it "funded."

Just an observation.  It's just another reason why we need better, centralized information on our not-for-profits' ALS research investment portfolios.

A Speeding Ticker Might Add An Interesting Dimension Next Year

There will be around 24 hours of scientific presentations over the next three days in the big global ALS MND symposium in Milan.

What if there were a ticker running under every Powerpoint presentation showing the names of the people who have died from ALS MND since last year's symposium presentations.

Would that be distracting?  Darned right it would be.  It should be.

So how fast would that ticker have to run?

Around 133,333 global deaths in a year / 24 hours = around 5,556 names per hour...

Around 5,556 names per hour / 60 minutes per hour / 60 seconds per minute = around 1.5 names per second.

Around 1.5 names per second moving relentlessly through all the scientific presentations and promise... That speeding ticker will be a measure that won't be pleasant to watch.  It's time we watched.

Dove Sono I Pazienti?

The years pass and the problems persist.

http://als-advocacy.blogspot.com/2012/12/look-whos-not-listening.html

http://als-advocacy.blogspot.com/2012/12/watch-out-shes-gonna-blow.html

Almost a thousand ALS MND organization employees and scientists and clinicians are meeting in Milan for the big annual symposium this week.  The format, accessibility, and fees continue to insulate them from the very patients and caregivers whose lives are at stake.

So far this week we saw the rollout of a twitter campaign to #shoutloud about ALS.  My hopes were up that it was about raising awareness of the global problem with those who are unaware.  It was disappointing that the twitter campaign wasn't designed to engage journalists and drug developers and activists and scientists and celebrities.  It was yet another ALS social media fundraiser.  http://www.mndfundraising.asn.au/event/shoutloudforalsandmnd/

Yesterday they had the "Ask the Experts" session, and I'm grateful for the livestream (although the time and link were a bit fluid until the last minute).  The experts were interesting.  It was more of a "Listen to the Experts" session, but it was informative.  I have a feeling that if the room had been full of people with ALS that the doc who ended his presentation with what was supposed to be a cute "it could always be worse" ending might have rethought that one.

Today the scientific presentations begin and we continue to have a virtual moat around the conference hotel.  Thanks to the few tweeters who move past pep-rally tweets and send us their insights into interesting content.

Every day people are diagnosed with ALS and they and their loved ones scramble to find out the most promising (and practical) ways to proceed.  It's hard to sort out.  This week we have the most renowned scientists discussing the most "promising" science, and those people with ALS and their loved ones are on on the outside, waiting for information through the lens of someone else.  It's nuts.  And it's nuts that people working on ALS science aren't hearing from directly from patients and seeing patients and learning from patients.

We live in a world that talks about the importance of patient-centricity.  Talk's cheap.